A drug to treat a very rare blood disorder affecting around 200 people in England has been recommended by the National Institute for Health and Care Excellence in further draft guidance.  

Produced by Alexion, eculizumab (Soliris) treats atypical Haemolytic Uraemic Syndrome (aHUS), an extremely rare but life-threatening disease which causes inflammation of blood vessels and the formation of blood clots throughout the body. People with aHUS are at constant risk of sudden and progressive damage to, and failure of vital organs, particularly the kidneys.

Eculizumab is being evaluated as part of a new programme at NICE that looks at highly specialised technologies which may benefit people with very rare diseases at a high price.

NICE’s estimates that this drug will cost the NHS up to £58 million in the first year, rising to £82 million after 5 years. It is currently funded by NHS England through interim specialised commissioning arrangements. 

As well as having a significant negative impact on quality of life, the prognosis for people with aHUS is poor, with around 10% to 15% of people with the disease dying in the initial, acute phase. The majority of people with aHUS – up to 70% – develop end stage kidney failure requiring dialysis. One patient in 5 has aHUS affecting organs other than the kidneys, most commonly the brain or heart.

For more details, go to: https://www.nice.org.uk/News/Press-and-Media/nice-draft-guidance-recommends-eculizumab-soliris-for-treatment-of-very-rare-life-threatening-blood-disorder